FDA Approves 3 Cystic Fibrosis Drugs of Vertex for Patients with Rare Mutations

On December 21st, Vertex Pharmaceuticals Incorporated announced that the FDA expanded the eligibility for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to TRIKAFTA, based on in vitro data.

SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) were also approved to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. These approvals allow more than 600 people with CF not previously eligible for these medicines an opportunity to potentially benefit from treatment that targets the underlying cause of their disease. These modulators rectify CFTR protein defects that result from certain CFTR mutations.

The approval for expanded use of three of our CF medicines based on our well-established in vitro model is a testament to the relentless commitment of our scientists to reach our goal of developing treatments for all people with CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex.

This use expansion came 3 months after Vertex applied for their label expansions.

Cystic Fibrosis

Cystic Fibrosis (CF) affects ~70,000 people worldwide with 1,000 new diagnoses per year. This disease results in a life expectancy of 42-50 years for those fortunate enough to live in the developed world. This increase in lifespan, despite being over 20 years below the average, is a testament to the scientific progress of recent decades. In the mid-20th century, most people born with the disease would not survive to reach puberty.

This condition is caused in 2/3rds of cases by an autosomal recessive mutation in the CFTR gene that controls the viscosity of various bodily fluids. There are ~1500 other mutations that can also be responsible for the disease in afflicted individuals. This vast collage of mutations that could be responsible for CF makes Vertex’s recent approvals seem like a comparative drop in the bucket despite the evident value they have for those 600 patients.

This mutation’s result is the loss of the amino acid phenylalanine and symptoms affecting not just the lungs but also the pancreas, reproductive organs (in males), and digestive tract.

The 3 Medications

TRIKAFTA was previously approved for people with at least one F508del mutation and is now approved for 177 additional mutations; SYMDEKO is now approved for 127 additional mutations, for a total of 154 SYMDEKO-responsive mutations; and KALYDECO is now approved for an additional 59 mutations, for a total of 97 KALYDECO-responsive mutations. In addition, for certain people with CF who are currently eligible for KALYDECO, this approval allows them to also be eligible for SYMDEKO or TRIKAFTA; and similarly, for those who are currently eligible for SYMDEKO, this approval allows them to also be eligible for TRIKAFTA.

Market Opportunity

With the global numbers of CF sufferers being relatively small, the authorization of medications for younger age groups has a lot of potential for Vertex. With approximately half of CF sufferers being below the age of 18, expanding the ability of medications to treat more patients is not only an opportunity for more customers but for Vertex’s public image.

While there are only a total of 600 new patients who can benefit from these previously produced drugs, the marginal cost of treating them is just the cost of manufacturing and the legal costs that got the drugs approved for these new patients in the first place. This is a profitable but relatively small-scale endeavor.

By Eduardo Longoria

Related Article: Vertex Bags EU Approval for Cystic Fibrosis Treatment in Children Aged 6-11 Years

References

https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-fda-approvals-trikaftar

Author

Eduardo Longoria